Basic Laboratory Research at UC Leads to Phase I Clinical Trial for Cancer

After decades of laboratory research and promising findings, the discoveries of Xiaoyang Qi, PhD, a University of Cincinnati College of Medicine and Cincinnati Cancer Center researcher and Bexion's scientific co-founder, are being translated to clinical trials in humans, possibly helping thousands living with cancer.

On, July 18, Bexion Pharmaceuticals LLC announced that the U.S. Food and Drug Administration cleared their application for a first-in-human Phase I clinical trial with the compound BXQ-350 for treatment of advanced solid tumors and glioblastoma multiforme, the most common type of brain cancer. 

"This is an exciting announcement as this is the goal of every scientist—to actually make a difference in patient care," says Qi, a member of the UC Cancer Institute and the UC Gardner Neuroscience Institute and Brain Tumor Center. "Over the years when I've published new research, e-mails from patients and family members asking about a clinical trial involving my findings hit my inbox. I'm so happy that now, I can answer back that there is one available."

In 2013, Bexion received a $2.9 million Small Business Innovation Research Bridge Award from the National Cancer Institute, with Qi as co-principal investigator, to help it bring BXQ-350 into the clinical trial phase.

The treatment is based on research from Qi's lab that began in the 1990s in his lab at Cincinnati Children's Hospital Medical Center. 

Qi, now a professor in the division of hematology oncology at UC, discovered SapC-DOPS, the combination of a lysosomal protein saposin C (SapC), and a phospholipid, known as dioleoylphosphatidylserine (DOPS), that assembled into tiny cavities, or nanovesicles, can target and kill many forms of cancer cells.

Lysosomes are membrane-enclosed cellular organelles that contain enzymes capable of breaking down all types of biological components; phospholipids are major components of all cell membranes and form lipid bilayers—or cell membranes.

Qi says his lab found that the combination of these two natural cellular components, or SapC-DOPS, caused cell death in human cancer cell types, including brain, lung, skin, prostate, blood, breast and pancreatic cancer, while sparing normal cells and tissues in animal models of human cancer. 

With numerous basic studies under his belt, Qi formed a partnership with Bexion to create the BXQ-350 compound which will now be tested in a Phase I clinical trial.

"Phase I trials help us determine acceptable dosages and possible side-effects," says John Morris, MD, director of the UC Cancer Institute's Phase I Experimental Therapeutics Program where the trial will be housed. Morris, also a faculty member at the UC College of Medicine and a member of the Cincinnati Cancer Center, is principal investigator on the clinical trial at UC, which is one of several sites hosting the study. 

"Phase 1 clinical research trials are the first step in moving tested scientific concepts from the laboratory bench to the patient; they typically include less than 50 people and are important for patients who may not have standard treatment options, as these therapies are novel and not widely available," he says. "The UC Cancer Institute is the only facility in the Tristate area with a Phase I program."

The trial is now being reviewed by the Institutional Review Board, and Morris says he hopes to enroll the first patient in September.